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The Value of Pharmaceuticals and Adherence to Therapy in the Management of Hematologic Cancers

February 2015 Vol 8, Special Issue: Payers' Perspectives in Oncology - Payers' Perspectives

Consistent with past meetings, the 2014 annual meeting of the American Society of Hematology (ASH) included very insightful and educational presentations on the value of pharmaceuticals, cost implications, and the importance of adherence to therapy that can help guide policy decisions for pharmacy and medical directors. This is especially impor­tant considering the impact of the new treatments for hematologic cancers that have been introduced to the market over the past 12 months.

Novel therapies for hematologic cancers are a major area of focus for payers. In 2014, the US Food and Drug Administration approved 4 new molecular entities for the treatment of hematologic cancers, impacting the medical and pharmacy budgets for payers. These 4 drugs include belinostat for relapsed or refractory peripheral T-cell lymphoma; blinatumomab for Philadelphia chromosome–negative relapsed or refractory precursor acute lymphoblastic leukemia; ibrutinib for several indications, including mantle-cell lymphoma and chronic lymphocytic leukemia (CLL); and idel­alisib for several indications, including CLL, follicular B-cell non-Hodgkin lymphoma, and small lymphocytic lymphoma.1 Of these novel therapies, 2 are administered intravenously (belinostat and blinatumomab) and 2 are administered orally (ibrutinib and idelalisib), with direct implications for payers.

Cancer care, including hematologic cancers, is a major area of focus in the current drug development arena, and is one of the leading contributors to the overall growth in spending for payers. This highlights the importance of evaluating the information presented at the recent ASH meeting as well as other developments in hematologic cancers.

Reimbursement, Value, and Cost-Effectiveness
At a special symposium on quality care and discussed in this special issue (see article on page 1), Andreas Laupacis, MD, MSc, suggested that drug reimbursement decisions should incorporate value and cost-effectiveness. Dr Laupacis is actively involved in drug reimbursement committee decisions in Canada. Focusing on cost-effectiveness, Canadian drug reimbursement committees decide whether to pay for particular drugs, decline to pay because of a lack of cost-effectiveness, or to pay for a restricted indication using public funds.

Even though we do not have nationalized healthcare in the United States, Dr Laupacis suggests that private payers in the United States can still dictate their reimbursement policies based on reliable cost-effective analyses. His suggestions for payers include:

  • Focusing on getting high-quality information about the effectiveness of drugs to accurately drive cost-effectiveness ratios
  • Negotiating drug prices aggressively based on cost-effectiveness
  • Using tools such as value-based discounts
  • Involving patients in the care dialogue.

Given these suggestions, and considering that more than $1 billion in additional funding was appropriated to comparative effectiveness research in the Affordable Care Act,2 we are likely to see more focus on value in healthcare, cost-effectiveness, and comparative effectiveness research in the near future.

Economic Burden and Adherence to Therapy
Several presentations at ASH 2014 focused on the cost of care for hematologic cancers and the impact of these costs on patient medication adherence. Decreased adherence to therapy can have a detrimental effect on patient morbidity and mortality, especially for cancer diagnoses.

Joanne S. Buzaglo, PhD, studied the economic burden on patients diagnosed with chronic myeloid leukemia (CML). The availability of new breakthrough therapies for the treatment of CML has transformed this condition from an often fatal disease to a chronic disease that requires ongoing treatment. The Cancer Support Community, in partnership with the Leukemia & Lymphoma Society, registered 484 patients living with CML to the Cancer Experience Registry. Overall, 81% of patients responded to a survey about the financial burden of CML and cancer-related psychological distress. Among these patients with CML, 19% reported missing a dose of their oral CML medication at least once monthly, 14% reported postponing prescriptions, and 10% said they skipped dosages of their prescribed CML medication. By these measures, 31% of the patients with CML were defined as having poor medication adherence (see article on page 1).

In addition, nearly 50% of the patients reported out-of-pocket (OOP) costs of ≥$100 related to CML, 27% spent ≥$250 monthly, 15% spent ≥$500, and 5% of patients spent ≥$1000. To be able to pay for their CML medication, 38% of patients said that they had to use copay assistance, 35% had to cut grocery expenses, 33% depleted their savings, 30% used pharmaceutical assistance programs, 18% sold personal property, 17% asked their healthcare provider for a less expensive treatment, 13% had liquidated their assets, 6% went into bankruptcy, and 4% of patients foreclosed on their homes. Other studies have also shown that cancer treatments can lead to rates of bankruptcy that are more than double the rate among people without cancer.3

These statistics are very concerning, and Dr Buzaglo concluded that “implications for future research and practice so that patients fully benefit from CML therapy include the development and evaluation of interventions that enhance patient–clinician communications, psychosocial distress screening and referral, and financial counseling and assistance.”

S. Yousuf Zafar, MD, MHS, who had coined the term “financial toxicity,” has been pointing at different occasions how the cost of paying for cancer impacts the efficacy of treatment. At ASH 2014, Dr Zafar said that a high patient cost burden is associated with a 70% higher likelihood of nonadherence to treatment. He stated that financial toxicity is as important as drug toxicity when it comes to the management of patients with cancer (see article on page 10).

Some of the statistics supporting the concept of “financial toxicity” in regard to the treatment of cancer include that 50% of Medicare beneficiaries pay 10% of the cost of OOP therapy, 28% of Medicare beneficiaries spend >20% of the cost of OOP treatment, and nearly 50% of patients take money from their savings and cut back on basics to pay for care.

In prospective, longitudinal studies of patients with cancer, Dr Zafar and colleagues found that 52% of patients want to discuss treatment-related OOP costs with their oncologist, and 51% want their oncologist to take cost into account when making treatment decisions, but only 19% of patients have actually discussed this with their oncologist, leaving a gap in treatment that can result in patient nonadherence.

By contrast, among patients who did discuss costs with their oncologist, 57% reported having lower OOP costs, primarily a result of being referred to a financial assistance organization. This is very interesting, considering that financial assistance programs and organizations contribute millions of dollars in aid annually to patients who cannot afford their medications. Specialty pharmacies are particularly adept at helping patients obtain this financial support when needed.

In addition to presenting data on the value of pharmaceuticals and adherence to therapy in the treatment of hematologic cancers, many studies were presented at ASH 2014 related to new advances in the treatment of hematologic cancers, advances in treatment regimens, and updates on the cost of treatment. ASH should be commended for another insightful and educational meeting.

1. CenterWatch. FDA approved drugs. Accessed February 6, 2015.
2. Donnelly J. Health policy briefs: Comparative effectiveness research. Health Affairs. Updated October 8, 2010. Accessed February 6, 2015.
3. Ramsey S, Blough R, Kirchoff A, et al. Washington State cancer patients found to be at greater risk for bankruptcy than people without a cancer diagnosis. Health Aff (Millwood). 2013;32:1143-1152.

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Last modified: August 30, 2021